Schärer Orlando D
Department of Pharmacological Sciences, Stony Brook University, Stony Brook, NY 11974-3400, USA.
DNA Repair (Amst). 2008 Feb 1;7(2):339-44. doi: 10.1016/j.dnarep.2007.10.007.
Alterations in genes involved in nucleotide excision repair (NER) are associated with three genetic disorders, xeroderma pigmentosum (XP), Cockayne syndrome (CS) and trichothiodystrophy (TTD). The transcription and repair factor TFIIH is a central component of NER and mutations of its subunits are associated with all three diseases. A recent report provides a molecular basis for how mutations in the NER endonuclease XPG that affect the interaction of TFIIH might give rise to CS features. In cells of XP-G patients with a combined XP and CS phenotype, XPG fails to associate with TFIIH and as a consequence the CAK subunit dissociates from core TFIIH. A simplified, but general model of how various assembly and disassembly states of TFIIH can be invoked to explain different disease states is discussed. Accordingly, defects in specific enzymatic functions typically result in XP, dissociation of the CAK subunit from TFIIH is associated with XP/CS and a more generalized destabilization of TFIIH gives rise to TTD. While this classification provides a useful framework to understand how alterations in TFIIH correlate with disease states, it does not universally apply and relevant exception and alternative explanations are discussed.
参与核苷酸切除修复(NER)的基因改变与三种遗传性疾病相关,即着色性干皮病(XP)、科凯恩综合征(CS)和毛发硫营养不良(TTD)。转录和修复因子TFIIH是NER的核心组成部分,其亚基的突变与所有这三种疾病都有关联。最近的一份报告为影响TFIIH相互作用的NER核酸内切酶XPG中的突变如何导致CS特征提供了分子基础。在具有XP和CS联合表型的XP - G患者的细胞中,XPG无法与TFIIH结合,结果CAK亚基从核心TFIIH上解离。本文讨论了一个简化但通用的模型,该模型可用于解释如何通过TFIIH的各种组装和拆卸状态来解释不同的疾病状态。因此,特定酶功能的缺陷通常会导致XP,CAK亚基从TFIIH上解离与XP/CS相关,而TFIIH更广泛的不稳定则会导致TTD。虽然这种分类为理解TFIIH的改变如何与疾病状态相关提供了一个有用的框架,但它并不普遍适用,本文还讨论了相关的例外情况和其他解释。